Identifier |
20210222_nanos_sciplatform2_03-video |
Title |
Assessment of rAAV2/2-ND4 Gene Therapy Efficacy in LHON Using an External Control Group |
Creator |
Nancy J. Newman; Patrick Yu-Wai-Man; Valerio Carelli; Mark Moster; Catherine Vignal-Clermont; Alfredo Sadun; Chiara La Morgia; Francesco Bandello; Lorena Castillo Campillo; Stéphanie Leruez; Dean Cestari; Rod Foroozan; Rustum Karanija; Laure Blouin; Michel Roux; Magali Taiel; José-Alain Sahel |
Affiliation |
(NJN) Emory University School of Medicine, Atlanta, GA; (PYWM) Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Cambridge, London, United Kingdom of Great Britain and Northern Ireland; (VC) (CLM) Unit of Neurology, Department of Biomedical and Neuromotor Sciences, Bologna, Italy; (MM) Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA; (CVC) Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France; (AS) Doheny Eye Institute/ UCLA School of Medicine, Los Angeles, CA; (FB) Department of Ophthalmology Vita-Salute San Raffaele University, Milan, Italy; (LCC) Institut Catala de Retina, Barcelona, Spain; (SL) CHU Angers - Hopital Hotel Dieu, Angers, France; (DC) Massachusetts Eye and Ear Infirmary, Boston, MA; (RF) Alkek Eye Center, Houston, TX; (RK) Doheny Eye Institute / UCLA School of Medicine, Los Angeles, CA; (LB) (MR) (MT) GenSight Biologics, Paris, France; (JAS) Institut de la Vision, Paris, France; Pittsburgh School of Medicine, Pittsburgh, PA |
Subject |
Optic Neuropathy; Genetic Disease |
Description |
The two Phase-3 multi-center clinical trials RESCUE and REVERSE showed sustained bilateral improvement of best-corrected visual acuity (BCVA) following unilateral injection of rAAV2/2-ND4 gene therapy for Leber Hereditary Optic Neuropathy (LHON). This bilateral treatment effect was compared to an external control group comprised of untreated ND4-LHON patients. |
Date |
2021-02 |
Language |
eng |
Format |
video/mp4 |
Type |
Image/MovingImage |
Source |
2021 North American Neuro-Ophthalmology Society Annual Meeting |
Relation is Part of |
NANOS Annual Meeting 2021: Scientific Platform Session II |
Collection |
Neuro-ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu |
Publisher |
Spencer S. Eccles Health Sciences Library, University of Utah |
Holding Institution |
North American Neuro-Ophthalmology Association. NANOS Executive Office 5841 Cedar Lake Road, Suite 204, Minneapolis, MN 55416 |
Rights Management |
Copyright 2021. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
ARK |
ark:/87278/s6xh5qck |
Context URL |
The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/ |
Setname |
ehsl_novel_nam |
ID |
1697222 |
Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6xh5qck |